Spastic Paraplegia, HereditaryPhase 2SPA-MNCT02314208
Therapeutic Metabolic Intervention in Patients With Spastic Paraplegia SPG5
A Phase 2 study testing Xenbilox, Resveratrol and Tahor in people with Spastic Paraplegia, Hereditary, run by Institut National de la Santé Et de la Recherche Médicale, France at 1 site in France.
Trial summary
The purpose of this project is to study the efficacy of three candidate molecules (Xenbilox, Tahor and Resveratrol) in order to decrease the production of oxysterols by reducing the synthesis of cholesterol and/or regulate the production of bile acids and/or enabling neuroprotective action within the motor neuron. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Spastic Paraplegia, Hereditary
- What is being tested?
- Xenbilox, Resveratrol and Tahor
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteFrance
- Study dates
- 8 Jan 2015to 1 Jan 2018
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- patients that have confirmed through genetic testing their status as carriers of 2 mutations in the CYP7B1 gene
- age ≥ 18 years
- patients that have signed the informed consent form
- presence of health care coverage
Who cannot join
- known hypersensitvity to chenodeoxycholic acid, atorvastatin, resveratrol or to any of their byproducts
- cholesterol lowering medications other than the study treatment
- hepatic failure with transaminases >3 times the normal level
- progressive biliary pathology
- chronic diarrhea
- serious mental illness
Show all 15Show fewer
- significant comorbid neurological disorder
- incapacity to understand information about the protocol
- unwilling or unable to participate in any part of the study
- participation in another clinical trial during the study period
- person deprived of liberty by judicial or administrative decision
- adult subject under legal protection or unable to consent
- pregnant or breastfeeding women
- lack of health care coverage
- absence of a signed informed consent form
Trial locations and contacts
1 site in 1 country.
About this study
The purpose of this project is to study the efficacy of three candidate molecules (Xenbilox, Tahor and Resveratrol) in order to decrease the production of oxysterols by reducing the synthesis of cholesterol and/or regulate the production of bile acids and/or enabling neuroprotective action within the motor neuron.
Read the detailed description
The primary objective of the study is:
- decrease the accumulation of metabolites which can have a negative impact on neurological and systemic function of patients with SPG5.
The secondary objectives of the study are:
- confirm the clinical and biological tolerance of the different candidate molecules under study
- improve the serum bile acid profile of patients with SPG5
- Conditions
- Spastic Paraplegia, Hereditary
- Being tested
- Xenbilox, Resveratrol, Tahor
- Keywords
- spastic paraplegia, metabolism, oxysterols, neurogenetics
- Medical subject headings
- Spastic Paraplegia, Hereditary, Paraplegia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
XenbiloxDrug
Also called Chenodeoxycholic acid
ResveratrolDietary supplement
TahorDrug
Also called Atorvastatin
| Study group | Role | What they receive |
|---|---|---|
| Xenbilox | Active comparator | Xenbilox (chenodeoxycholic acid) 1000mg capsule by mouth every day for 2 months Receives: Drug: Xenbilox |
| Resveratrol | Active comparator | Resveratrol 80mg capsule by mouth every day for 2 months Receives: Dietary Supplement: Resveratrol |
| Tahor | Active comparator | Tahor (atorvastatin) 40mg tablet by mouth every day for 2 months Receives: Drug: Tahor |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change of 27-hydroxycholesterol blood level after 2 months of treatment for each treatment
27-hydroxycholesterol will be measured on fasten blood samples for the 12 patients included before and after the 2-month treatment period for each treatment
Measured: 2 months
Secondary outcomes (4)
Modification of the serum acid profile after a 2-month treatment measured in blood and urine samples
In blood and urines samples of the 12 patients, the following criteria will be assesed in order to observe an evolution of the acid profile:
- total biliary acids
- ratio of primary/secondary biliary acids
- the ratio cholic acids/chenodeoxycholic acids
- the ratio chenodeoxycholic acids/ lithocholic acids
Measured: 2 months
Change of 25-hydroxycholesterol blood level after 2 months of treatment for each treatment
25-hydroxycholesterol will be measured on fasten blood samples for the 12 patients included before and after the 2-month treatment period for each treatment
Measured: 2 months
Number of participants with adverse events related with digestive problems
During the study, adverse events related with digestive problems will be evaluate in order to asses the clinical tolerance to each treatment
Measured: 18 months
Number of partcipants with adverse events related with standard biological parameters
Biological tolerance will be evaluate on patients blood samples at each visit. The following criteria will be assessed:
- ionogram and fasting glucose
- kidney and liver function with urea, creatinine,lipase, amylase, bilirubine, creatine phosphokinase, transaminases,sodium, potassium, calcium, phosphorus
Measured: 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Dec 2014 (estimated)
- Study start8 Jan 2015
- Primary completion27 Sep 2017
- Study completion1 Jan 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Spastic Paraplegia, HereditaryStudies of the same condition, open and under way.
- More trials run by Institut National de la Santé Et de la Recherche Médicale, FranceThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Sponsor type
- Other government
- Responsible party
- Sponsor
- Investigators
- Fanny MOCHEL, MD-PhD, Principal investigator, Pitié-Salpêtrière Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02314208
- Sponsor's protocol ID
- C14-04
- Acronym
- SPA-M
- Status last verified
- Feb 2025
- First submitted
- 14 Nov 2014
- Last updated
- 2 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
Publications
Marelli C, Lamari F, Rainteau D, Lafourcade A, Banneau G, Humbert L, Monin ML, Petit E, Debs R, Castelnovo G, Ollagnon E, Lavie J, Pilliod J, Coupry I, Babin PJ, Guissart C, Benyounes I, Ullmann U, Lesca G, Thauvin-Robinet C, Labauge P, Odent S, Ewenczyk C, Wolf C, Stevanin G, Hajage D, Durr A, Goizet C, Mochel F. Plasma oxysterols: biomarkers for diagnosis and treatment in spastic paraplegia type 5. Brain. 2018 Jan 1;141(1):72-84. doi: 10.1093/brain/awx297.
PubMed 29228183
Study at a glance
From the registry record, last updated by the sponsor on 2 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Therapeutic Metabolic Intervention in Patients With Spastic Paraplegia SPG5
- ClinicalTrials.gov ID
- NCT02314208
- Acronym / sponsor ID
- SPA-M / C14-04
- Conditions
- Spastic Paraplegia, Hereditary
- Keywords
- spastic paraplegia, metabolism, oxysterols, neurogenetics
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Institut National de la Santé Et de la Recherche Médicale, FranceOther government
- Study start
- 8 Jan 2015 (actual)
- Primary completion
- 27 Sep 2017 (actual)
- Study completion
- 1 Jan 2018 (actual)
- First posted
- 11 Dec 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this project is to study the efficacy of three candidate molecules (Xenbilox, Tahor and Resveratrol) in order to decrease the production of oxysterols by reducing the synthesis of cholesterol and/or regulate the production of bile acids and/or enabling neuroprotective action within the motor neuron.
Detailed description
The primary objective of the study is:
- decrease the accumulation of metabolites which can have a negative impact on neurological and systemic function of patients with SPG5.
The secondary objectives of the study are:
- confirm the clinical and biological tolerance of the different candidate molecules under study
- improve the serum bile acid profile of patients with SPG5
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Xenbilox | Active comparator | Drug: XenbiloxXenbilox (chenodeoxycholic acid) 1000mg capsule by mouth every day for 2 months |
| Resveratrol | Active comparator | Dietary Supplement: ResveratrolResveratrol 80mg capsule by mouth every day for 2 months |
| Tahor | Active comparator | Drug: TahorTahor (atorvastatin) 40mg tablet by mouth every day for 2 months |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- patients that have confirmed through genetic testing their status as carriers of 2 mutations in the CYP7B1 gene
- age ≥ 18 years
- patients that have signed the informed consent form
- presence of health care coverage
Key exclusion criteria (15 listed)
- known hypersensitvity to chenodeoxycholic acid, atorvastatin, resveratrol or to any of their byproducts
- cholesterol lowering medications other than the study treatment
- hepatic failure with transaminases >3 times the normal level
- progressive biliary pathology
- chronic diarrhea
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| XenbiloxAlso: Chenodeoxycholic acid | Drug | Xenbilox |
| Resveratrol | Dietary supplement | Resveratrol |
| TahorAlso: Atorvastatin | Drug | Tahor |
Primary outcome
Change of 27-hydroxycholesterol blood level after 2 months of treatment for each treatment
Time frame: 2 months
27-hydroxycholesterol will be measured on fasten blood samples for the 12 patients included before and after the 2-month treatment period for each treatment
4 secondary outcomes
Modification of the serum acid profile after a 2-month treatment measured in blood and urine samples
Time frame: 2 months
Change of 25-hydroxycholesterol blood level after 2 months of treatment for each treatment
Time frame: 2 months
Number of participants with adverse events related with digestive problems
Time frame: 18 months
Number of partcipants with adverse events related with standard biological parameters
Time frame: 18 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Sep 2025.
- Results publication: PubMed 29228183Marelli C, Lamari F, Rainteau D, Lafourcade A, Banneau G, Humbert L, Monin ML, Petit E, Debs R, Castelnovo G, Ollagnon E, Lavie J, Pilliod J, Coupry I, Babin PJ, Guissart C, Benyounes I, Ullmann U, Lesca G, Thauvin-Robinet C, Labauge P, Odent S, Ewenczyk C, Wolf C, Stevanin G, Hajage D, Durr A, Goizet C, Mochel F. Plasma oxysterols: biomarkers for diagnosis and treatment in spastic paraplegia type 5. Brain. 2018 Jan 1;141(1):72-84. doi: 10.1093/brain/awx297.
Related trials for Spastic Paraplegia, Hereditary
Active, not recruiting
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis
Active, not recruiting
Flexibility, Resistance, Aerobic, Movement Execution Training in Adults With Hereditary Spastic Paraplegia
Hereditary Spastic Paraplegia
RecruitingObservational study
Rare Disease Registry Database for Movement Disorders in Southern Anhui Province
Motor Neuron Diseases, Spinocerebellar Ataxias
RecruitingObservational study
TreatHSP Platform: Adaptive Natural History Platform for Ataxias, HSPs, and Spastic Ataxias
Hereditary Spastic Paraplegia, Spastic Ataxia
RecruitingObservational study
A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders
AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Fanny MOCHEL, MD-PhD principal investigator
Pitié-Salpêtrière Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Xenbilox, Resveratrol and Tahor in people with Spastic Paraplegia, Hereditary, run by Institut National de la Santé Et de la Recherche Médicale, France at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change of 27-hydroxycholesterol blood level after 2 months of treatment for each treatment (time frame: 2 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02314208, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Sep 2025.